CN112574982B - 用于治疗高胱氨酸尿症的胱硫醚β-合酶 - Google Patents

用于治疗高胱氨酸尿症的胱硫醚β-合酶 Download PDF

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CN112574982B
CN112574982B CN202011103747.1A CN202011103747A CN112574982B CN 112574982 B CN112574982 B CN 112574982B CN 202011103747 A CN202011103747 A CN 202011103747A CN 112574982 B CN112574982 B CN 112574982B
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cbs
leu
gly
pegylated
glu
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CN112574982A (zh
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J·P·克劳斯
T·马吉坦
E·巴伯李尔
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University of Colorado Boulder
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/51—Lyases (4)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/19—Carboxylic acids, e.g. valproic acid
    • A61K31/195—Carboxylic acids, e.g. valproic acid having an amino group
    • A61K31/197—Carboxylic acids, e.g. valproic acid having an amino group the amino and the carboxyl groups being attached to the same acyclic carbon chain, e.g. gamma-aminobutyric acid [GABA], beta-alanine, epsilon-aminocaproic acid or pantothenic acid
    • A61K31/198—Alpha-amino acids, e.g. alanine or edetic acid [EDTA]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/205—Amine addition salts of organic acids; Inner quaternary ammonium salts, e.g. betaine, carnitine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/38—Heterocyclic compounds having sulfur as a ring hetero atom
    • A61K31/385—Heterocyclic compounds having sulfur as a ring hetero atom having two or more sulfur atoms in the same ring
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/56—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • A61P19/10—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/88—Lyases (4.)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/96—Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y402/00—Carbon-oxygen lyases (4.2)
    • C12Y402/01—Hydro-lyases (4.2.1)
    • C12Y402/01022—Cystathionine beta-synthase (4.2.1.22)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Dermatology (AREA)
  • Rheumatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Ophthalmology & Optometry (AREA)
  • Cardiology (AREA)
  • Vascular Medicine (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Pulmonology (AREA)
  • Neurology (AREA)
  • Urology & Nephrology (AREA)
  • Neurosurgery (AREA)
CN202011103747.1A 2013-01-29 2014-01-29 用于治疗高胱氨酸尿症的胱硫醚β-合酶 Active CN112574982B (zh)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201361758138P 2013-01-29 2013-01-29
US61/758138 2013-01-29
US13/803,804 US9034318B2 (en) 2011-08-30 2013-03-14 Chemically modified cystathionine beta-synthase enzyme for treatment of homocystinuria
US13/803804 2013-03-14
CN201480006554.0A CN105452457A (zh) 2013-01-29 2014-01-29 用于治疗高胱氨酸尿症的胱硫醚β-合酶
PCT/US2014/013602 WO2014120770A1 (en) 2013-01-29 2014-01-29 Cystathionine beta-synthase enzyme for treatment of homocystinuria

Related Parent Applications (1)

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CN201480006554.0A Division CN105452457A (zh) 2013-01-29 2014-01-29 用于治疗高胱氨酸尿症的胱硫醚β-合酶

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CN112574982A CN112574982A (zh) 2021-03-30
CN112574982B true CN112574982B (zh) 2025-05-13

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CN201480006554.0A Pending CN105452457A (zh) 2013-01-29 2014-01-29 用于治疗高胱氨酸尿症的胱硫醚β-合酶

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US (4) US9034318B2 (enExample)
EP (4) EP4406536A3 (enExample)
JP (4) JP6453243B2 (enExample)
CN (2) CN112574982B (enExample)
AU (3) AU2014212471B2 (enExample)
BR (1) BR112015017775A2 (enExample)
DK (1) DK3712264T5 (enExample)
FI (1) FI3712264T3 (enExample)
HK (1) HK1213291A1 (enExample)
IL (1) IL239783B (enExample)
PT (1) PT3712264T (enExample)
WO (1) WO2014120770A1 (enExample)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2003251550A1 (en) 2002-06-17 2003-12-31 The Regents Of The University Of Colorado HUMAN CYSTATHIONINE Beta-SYNTHASE VARIANTS AND METHODS OF PRODU CTION THEREOF
US9034318B2 (en) * 2011-08-30 2015-05-19 The Regents Of The University Of Colorado, A Body Corporate Chemically modified cystathionine beta-synthase enzyme for treatment of homocystinuria
US9675678B2 (en) 2013-01-29 2017-06-13 The Regents Of The University Of Colorado, A Body Corporate Compositions and methods for treatment of homocystinuria
EP3415489A1 (en) 2013-10-18 2018-12-19 Deutsches Krebsforschungszentrum Labeled inhibitors of prostate specific membrane antigen (psma), their use as imaging agents and pharmaceutical agents for the treatment of prostate cancer
ES2994256T3 (en) 2015-11-09 2025-01-21 Univ Colorado Regents Compositions and methods for treatment of homocystinuria
CA3059592A1 (en) 2017-04-17 2018-10-25 The Regents Of The University Of Colorado, A Body Corporate Optimization of enzyme replacement therapy for treatment of homocystinuria
CN110603324A (zh) 2017-05-12 2019-12-20 得克萨斯大学体系董事会 用于治疗高同型半胱氨酸血症和同型胱胺酸尿症患者的人类酶介导的同型半胱氨酸耗竭
US20190309271A1 (en) 2018-03-20 2019-10-10 Rubius Therapeutics, Inc. Therapeutic cell systems and methods for treating homocystinuria
CN108795913B (zh) * 2018-05-04 2021-07-02 山西大学 一种植物中能催化h2s产生的酶及其应用
CN113260324A (zh) 2018-10-26 2021-08-13 得克萨斯大学体系董事会 用于治疗用途的工程化灵长目动物胱氨酸/半胱氨酸降酶
EP3990005B1 (en) 2019-06-26 2025-08-27 Travere Therapeutics Switzerland GmbH Pegylated cystathionine beta synthase for enzyme therapy for treatment of homocystinuria
CA3152379A1 (en) * 2019-09-03 2021-03-11 Travere Therapeutics Switzerland Gmbh Cystathionine beta-synthase enzyme therapy for treatment of elevated homocysteine levels
USD1013704S1 (en) * 2021-07-09 2024-02-06 The Regents Of The University Of Colorado, A Body Corporate Display screen or portion thereof with graphical user interface
USD1014518S1 (en) * 2021-07-09 2024-02-13 The Regents Of The University Of Colorado, A Body Corporate Display screen or portion thereof with graphical user interface
CN115721735A (zh) * 2022-10-13 2023-03-03 呈诺再生医学科技(北京)有限公司 Cbs基因在制备糖尿病视网膜病变治疗药物中的应用

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CN1612937A (zh) * 2001-11-06 2005-05-04 凯奇股份有限公司 用于高半胱氨酸和胱硫醚的酶促循环测定
CN101322840A (zh) * 2008-05-28 2008-12-17 中国人民解放军军事医学科学院卫生学环境医学研究所 一种人胱硫醚β-合成酶重组蛋白及应用

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AU2003251550A1 (en) * 2002-06-17 2003-12-31 The Regents Of The University Of Colorado HUMAN CYSTATHIONINE Beta-SYNTHASE VARIANTS AND METHODS OF PRODU CTION THEREOF
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US9364497B2 (en) 2010-07-01 2016-06-14 Isis Innovation Limited Treatment of cognitive disorders
US9034318B2 (en) * 2011-08-30 2015-05-19 The Regents Of The University Of Colorado, A Body Corporate Chemically modified cystathionine beta-synthase enzyme for treatment of homocystinuria
US9243239B2 (en) 2012-03-26 2016-01-26 The Regents Of The University Of Colorado, A Body Corporate Purification of cystathionine beta-synthase
US9675678B2 (en) * 2013-01-29 2017-06-13 The Regents Of The University Of Colorado, A Body Corporate Compositions and methods for treatment of homocystinuria

Patent Citations (2)

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Publication number Priority date Publication date Assignee Title
CN1612937A (zh) * 2001-11-06 2005-05-04 凯奇股份有限公司 用于高半胱氨酸和胱硫醚的酶促循环测定
CN101322840A (zh) * 2008-05-28 2008-12-17 中国人民解放军军事医学科学院卫生学环境医学研究所 一种人胱硫醚β-合成酶重组蛋白及应用

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JP6453243B2 (ja) 2019-01-16
CN105452457A (zh) 2016-03-30
AU2022263598A1 (en) 2022-12-15
JP2016506727A (ja) 2016-03-07
US20180311325A1 (en) 2018-11-01
EP4406536A3 (en) 2025-01-01
DK3712264T5 (da) 2024-08-26
AU2014212471A8 (en) 2015-08-13
HK1249137A1 (en) 2018-10-26
EP3712264A1 (en) 2020-09-23
US20150344864A1 (en) 2015-12-03
AU2022263598B2 (en) 2025-06-05
EP3282013B1 (en) 2020-05-20
EP4406536A2 (en) 2024-07-31
FI3712264T3 (fi) 2024-05-07
US9447406B2 (en) 2016-09-20
US9034318B2 (en) 2015-05-19
WO2014120770A1 (en) 2014-08-07
JP2023062201A (ja) 2023-05-02
US10046036B2 (en) 2018-08-14
JP7580822B2 (ja) 2024-11-12
AU2020200719A1 (en) 2020-02-20
US20140212403A1 (en) 2014-07-31
BR112015017775A2 (pt) 2017-11-21
CN112574982A (zh) 2021-03-30
US10624959B2 (en) 2020-04-21
AU2014212471B2 (en) 2019-11-28
EP3712264B1 (en) 2024-04-10
JP2021000142A (ja) 2021-01-07
PT3712264T (pt) 2024-05-09
US20170065687A1 (en) 2017-03-09
IL239783B (en) 2019-10-31
HK1213291A1 (zh) 2016-06-30
JP7240743B2 (ja) 2023-03-16
IL239783A0 (en) 2015-08-31
EP3712264A8 (en) 2020-11-04
DK3712264T3 (da) 2024-05-06
EP3282013A1 (en) 2018-02-14
EP2951299A1 (en) 2015-12-09
EP2951299B1 (en) 2017-07-12
AU2014212471A1 (en) 2015-07-23
CA2898772A1 (en) 2014-08-07
JP2019059768A (ja) 2019-04-18
AU2020200719B2 (en) 2022-08-18

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